News on TFR

In this section, we are collecting and publishing news focused on treatment-free remission in CML on a regular basis. We offer you to check the "News on TFR" section to be informed about latest advances in stopping treatment for CML patients.

Abstract

BCR-ABL1 tyrosine kinase inhibitors have dramatically improved outcomes for patients with chronic myeloid leukemia, and current studies are investigating whether some patients may be able to suspend therapy yet maintain response in a state known as “treatment-free remission” (TFR). Results from ongoing studies suggest that ≈ 40% to 60% of patients in sustained (generally ≥ 2 years) deep molecular response (defined as a 4-log or deeper reduction in BCR-ABL1 transcripts, depending on the study) who attempt TFR may successfully remain off treatment. Results from TFR clinical trials, patient considerations for attempting TFR, and potential predictive factors associated with successful TFR are reviewed herein.

FDA updates the label of Tasigna to reflect that certain patients with a type of leukaemia that may be eligible to stop treatment after sustained response

Discontinuation in treatment marks a first in Chronic Myeloid Leukemia

FDA News Release. December 22, 2017.

The U.S. Food and Drug Administration has updated the product label for the cancer drug Tasigna (nilotinib) to include information for providers about how to discontinue the drug in certain patients. Tasigna, first approved by the FDA in 2007, is indicated for the treatment of patients with Philadelphia chromosome-positive (Ph+) chronic myeloid leukaemia (CML). With today’s updated dosing recommendations, patients with early (chronic) phase CML who have been taking Tasigna for three years or more, and whose leukaemia has responded to treatment according to specific criteria as detected by a test that has received FDA marketing authorization, may be eligible to stop taking Tasigna.

“Patients diagnosed with CML generally face a lifetime of treatment to keep their leukaemia from growing or recurring,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “This approval shows that some patients may be able to stop treatment with Tasigna altogether if they are showing a strong response to therapy. While we welcome this progress in patient care, it’s important to note that any discontinuation of treatment still means patients must be regularly monitored for disease recurrence.”

Chronic Myeloid Leukemia

also called: Chronic Myelogeneous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

Philadelphia chromosome

A certain change in chromosomes (on chromosome 22) found in 95% of patients who have CML. The Philadelphia chromosome results from a mutation that involves the fusion of parts of chromosome 9 and chromosome 22 (the bcr-abl fusion gene)

Chromosome

A chromosome is a structure of DNA that carries the genetic makeup in the nucleus of the cell. Chromosomes contain giant chain molecules of DNA, coiled and folded as aggregates with specific proteins. Chromosomes ensure that during cell division the hereditary information is evenly distributed to the daughter cells. Normal human body cells have 46 chromosomes. Cancer cells can have a different number and/or structure of chromosomes.

Nilotinib

Trade name: Tasigna, development name: AMN107, inhibits BCR-ABL tyrosine kinase. Authorized for marketing in the EU since 2007 for the treatment of CML and Ph+ALL.Other names: |AMN107|Tasigna

BCR-ABL

The abnormal gene that characterizes Chronic Myeloid Leukemia, which is a fusion of the BCR gene of chromosome 9 and the ABL gene of chromosome 22

Chronic

Long-lasting, slowly developping

Gene

A unit of information present as DNA; a gene usually contains the blueprint for a protein.

CML

Chronic Myeloid Leukemia, also called Chronic Myelogenous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

Ph+

Abbreviation for "Philadelphia-Chromosome-positive", meaning the presence of a certain change in chromosomes (on chromosome 22) found in 95% of patients who have CML. The Philadelphia chromosome results from a mutation that involves the fusion of parts of chromosome 9 and chromosome 22 (the bcr-abl fusion gene).

CHR

Abbreviation for Complete Hematologic Response. The blood cell count has returned to normal, and tests don’t show any immature white blood cells. Also, the spleen has returned to a normal size if it was enlarged.

FDA

An agency of the United States Department of Health and Human services. The FDA is responsible for protecting and promoting public health through the regulation and supervision of food and drugs.